Described herein are compositions, kits and methods for shredding the genomes of selected cell types, for example, the genomes of selected cancer cell types.

Described herein are compositions, kits and methods for shredding the genomes of selected cell types, for example, the genomes of selected cancer cell types.

Provided are methods, compositions, and cells for use in adoptive cell therapy for the treatment of cancer. The methods involve administering an effective amount of cells to a subject, wherein the cells are modified ex vivo to suppress endogenous Zbtb20 expression and/or activity within the modified cells. The cells may comprise a dominant negative Zbtb20 capable of suppressing endogenous Zbtb20 activity, at least one shRNA capable of suppressing endogenous Zbtb20 expression, or at least one sgRNA capable of suppressing endogenous Zbtb20 expression. The cells may further comprise an exogenous TCR and/or CAR suitable for treating cancer. The method can further involve administering one or more additional cancer therapies, such as cells which express at least one exogenous TCR and/or CAR suitable for treating cancer. The method can provide various advantages, such as a reduction and/or elimination of an amount of cancer cells in the subject.

Therapeutic methods and medicines may be developed by identifying a gene responsible for progressive supranuclear palsy, as may effective therapeutic methods and medicines. A medicine for progressive supranuclear palsy may contain a compound for inhibiting the expression of a filamin-A gene is provided. Also provided is an assessment system that uses cells expressing filamin-A, which is used in the search for medicaments for progressive supranuclear palsy or their candidates.

The present disclosure belongs to the field of biomedicine. Specifically, the present disclosure relates to a circular RNA molecule, a cyclization precursor RNA molecule, a recombinant nucleic acid molecule, a recombinant expression vector, a recombinant host cell, a composition and use thereof in targeted degradation of a protein of interest, as well as a method for preventing or treating a disease. The circular RNA molecule has good membrane permeability, is easily delivered into cells, and has high-efficiency in vivo protein targeted degradation activity. The circular RNA molecule of the present disclosure successfully achieves inhibition of tumor growth, which proves the in vivo protein degradation activity of bio-PROTACs for the first time, and provides a positive and effective treatment solution for the treatment of diseases such as tumor.

The present disclosure provides methods of treating subjects having psoriasis, and methods of identifying subjects having an increased risk of developing psoriasis.

The present disclosure provides methods of treating subjects having psoriasis, and methods of identifying subjects having an increased risk of developing psoriasis.

This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.

Disclosed herein are embodiments of a nanoparticle composition suitable for delivering a therapeutic agent to a subject by nebulization. The nanoparticle comprises an ionizable lipid, cholesterol or a derivative thereof, a structural lipid, and a PEG lipid. The nanoparticle may partially or completely encapsulate a therapeutic agent, and may be nebulized for administration, such as by inhalation. In some embodiments, the therapeutic agent is mRNA.

Disclosed herein are embodiments of a nanoparticle composition suitable for delivering a therapeutic agent to a subject by nebulization. The nanoparticle comprises an ionizable lipid, cholesterol or a derivative thereof, a structural lipid, and a PEG lipid. The nanoparticle may partially or completely encapsulate a therapeutic agent, and may be nebulized for administration, such as by inhalation. In some embodiments, the therapeutic agent is mRNA.